By Colin Kellaher


Regeneron Pharmaceuticals Inc. on Tuesday said the U.S. Food and Drug Administration granted priority review to its application seeking approval of pozelimab for the treatment of an ultra-rare and life-threatening hereditary immune disease known as Chaple.

The Tarrytown, N.Y., biotechnology company said the application covers pozelimab in patients ages one and older with Chaple, or CD55 deficiency with hyperactivation of complement, angiopathic thrombosis and protein losing enteropathy, for which there are currently no FDA-approved treatments.

Chaple causes overactivation of the complement system, leading to potentially life-threatening abdominal and cardiovascular symptoms. Regeneron said pozelimab is designed to block the activity of complement factor C5, a protein involved in complement system activation.

The FDA grants priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period. Regeneron said the agency has set a target action date in Aug. 20 for the application.


Write to Colin Kellaher at colin.kellaher@wsj.com


(END) Dow Jones Newswires

02-21-23 0750ET